Participating in Clinical Trials

Participating in Clinical Trials

The decision to participate in a clinical trial is one that you need to consider carefully. Educate yourself about the investigational drug as well as the clinical trial to assess:

Effectiveness and

safety profile

Safety Profile: An outline of the risks and potential negative side effects of a treatment that is weighed against the possible benefits and used to determine if a treatment will be made available.

of the investigational drug

Risks and benefits of trial participation

Trial participation requirements (e.g. how many times you need to visit the clinical site)

One of the best sources of information is your physician(s) as well as the trial site investigator.

It’s important to know that clinical trials are not treatments; they are research studies designed to answer very specific questions about a drug or procedure. When deciding to participate in a clinical trial you will need to weigh the potential trial benefits along with the associated risks.

Potential Benefits

Receive access to an investigational drug not currently available by a prescription

Receive medical care by an expert study team

Contribute to what is known about FOP

Advance the science around FOP

Help future people diagnosed with FOP by contributing to the development of a potential therapy

Potential Risks

You could experience unwanted side effects of the investigational medicine, and sometimes these can be serious or life-threatening

The investigational drug may not provide you with any benefit

You may be given a placebo and receive no investigational drug (although participants will usually receive the drug after the placebo portion of the trial is over)

Eligibility Requirements

If you are interested in participating in a clinical trial, one of the first questions you should ask is whether you are eligible for the trial. All clinical trials have inclusion and exclusion criteria that describe who can and cannot participate. These criteria are set up to:

Help ensure the safety of those participating

Help the study sponsor answer their main or primary study objective(s)

Many clinical trials start in adults first before studying the investigational drug in children, so it is very common to see age listed as an inclusion criterion. The level of FOP disease activity can also be a common clinical trial inclusion or exclusion criteria.

The Informed Consent Form

Before participating in a clinical trial, you (or your child) will be asked to sign an informed consent form. Informed consent is a process by which the trial site investigator educates you about:

Purpose of the clinical trial

Potential drug benefits

Potential risks and side effects associated with the study drug

What’s required from you (or your child) in order to participate

You should also be informed that you do not have to stay in the clinical trial and may leave the trial at any point.

These meetings are your opportunity to ask questions about the trial or the drug. You should never feel rushed during the informed consent process, and you should have all your questions answered to your satisfaction.

Remember that your participation in a clinical trial is always voluntary.